Analyst Conference Summary



conference date: February 8, 2018 @ 5:30 AM Pacific Time
for quarter ending: December 30, 2017 (fourth quarter, Q4)

Forward-looking statements

Overview: Preparing for Patisiran launch in 2018, pending approval.

Basic data (GAAP):

Revenue was $37.9 million, up sequentially from $17.1 million, and up from $17.5 million year-earlier. All revenue was from collaborations.

Net income was negative $142.2 million, down sequentially from negative $122.9 million, and down from negative $112.9 million year-earlier.

Diluted EPS was negative $1.48, flat sequentially from negative $1.34, and down from negative $1.32 year-earlier.


Cash balance at the end of 2018 expected near $1.0 billion.

Full year 2018 non-GAAP R&D expense $400 to $440 million; SG&A expense $280 to $320 million.

Conference Highlights:

John Maraganore, CEO of Alnylam, said: "We have initiated a staged build of global medical and commercial capabilities to expand our reach and ensure that patients and physicians are educated about this rare disease and the safety and efficacy profile of patisiran, upon market approval . . . In sum, we believe our efforts position the Company to achieve its Alnylam 2020 goals of building a multi-product, commercial-stage company with a deep clinical-stage pipeline and robust product engine by the end of 2020, a profile rarely achieved in biotech history."

Positive Phase 3 Apollo results and NDA to FDA for Patisiran for hereditary TTR-mediated amyloidosis with polyneuropathy (hATTR-PN), also known as familial amyloidotic polyneuropathy (FAP) should enable a commercial launch in the second half of 2018. PDUFA date is August 11.

All revenue was from collaborators: $20.1 million from The Medicines Company, $13.4 million from Genzyme/Sanofi, and $4.4 million from other sources. Some of this revenue resulted from payments for milestones achieved.

Cash and equivalents balance at the end of the quarter was $1.73 billion, up sequentially from $1.15 billion. Sold $784.5 million worth of stock in the quarter. Repaid $120 million of its term loan agreement. $30 million in long-term debt.

Non-GAAP net income negative $115.1 million, down sequentially from negative $97.0, and down from negative $92.3 million year-earlier. EPS negative $1.20, down sequentially negative $1.06 from , and down from negative $1.08 year-earlier. Excluded non-cash stock based compensation was $27 million.

Alnylam continued a Phase 1 trial for ALN-TTRsc02, an ESC-GalNAc-siRNA conjugate targeting TTR for the treatment of ATTR amyloidosis, which is expected to enable a once-quarterly subcutaneous dosing regimen. Alnylam now owns all the rights. A Phase 3 trial is expected to begin in late 2018. Believes this could be expanded to help carriers, which would greatly expand the patient pool.

Fitusiran for hemophilia and rare bleeding disorders Phase 3 ATLAS pivotal study (actually a set of 3) restarted after a suspension following a patient death. Sanofi Genzyme is a partner in the program and is acquiring Bioverativ, a hemophilia focused pharma company.

Givosiran (ALN-AS1) for acute hepatic porphyrias Phase 3 trial design approved by the FDA and the ENVISION trial was initiated. Interim data is expected mid-2018, with an NDA possible by the end of 2018.

ALN-GO1 for primary hyperoxaluria type 1 (PH1) Phase 1/2 study in Europe continued and updated positive data was presented.

ALF-F12 targeting factor XII is now in development for the treatment of hereditary angioedema and for thromboprophylaxis.

Inclisiran (ALN-PCSsc), being developed by The Medicines Company, for hypercholesterolemia, started its Phase 3 trial, with complete enrollment expected soon. Alnylam could receive milestones and up to 20% royalties.

Cemdisiran (ALN-CC5) for aHUS (atypical hemolytic-uremic syndrome) started a Phase 2 study.

ALN-HBV Phase 1 study continued.

See also Alnylam pipeline.

Operating expenses of $185 million consisted of: $118 million for research and development and $67 million for general and administrative expense. Interest & other income was $0 million. Income taxes $0 million.

Alnylam hopes to begin commercial sales of Patisiran in 2018, Fitusiran in 2019, and Givosiran in 2020.


Manufacturing plans? For Patisiran we are in good shape for the U.S. and Europe, which we will expand to the rest of world. Inspections are going well.

Timeline for Atlas studies? After hold was lifted, enrollment should resume soon, we hope to get data in the 2019 time frame.

Givosiran sample size adjustment? It was always in the plan, we just had not provided guidance on it. It is not data driven.

Pfizer TTR candidate? The have a very large study running. We look forward to hearing the results. We are in good shape to compete with Patisiran. Their TTR stabilization is a pretty weak approach. TTR lowering is substantially better.

Patisiran breadth of label? We hope we can achieve a broad label based on the data, the symptoms treated, and the secondary data. We believe the patients studied were representative of the general TTR population. Apollo data was robust and consistent across all aspects of the disease. A number of Patisiran patients have shown disease reversal.

Last year we chose to focus on late stage executions rather than new INDs. But more programs will come forward using our new technology.

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Disclaimer: My analyst call summaries may include both our condensations of statements made by company representatives and my own analysis. They are not covered by any warranty. I cannot guarantee anything said by company representatives is true. I try not to make errors, but it is possible. This is investment journalism, not financial advice.

Copyright 2018 William P. Meyers